Research Studies Finder

Use this finder to find a study or trial that is the right fit for your child. Also, filter results by phase — which are the specific steps researchers take throughout the trial to find answers, solutions and cures.

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Showing 1 - 10 of 324 results

Uproleselan in Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS) or Mixed Phenotype Acute Leukemia (MPAL)

This study enrolls patients that have been diagnosed with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or mixed phenotype acute leukemia (MPAL) that has either come back (‘relapsed’) or does not respond to therapy (‘refractory’). This study involves taking a drug called uproleselan that is not yet approved for use in children or adults. The purpose of the study is to find a safe dose of the study drug in children that can be given with standard chemotherapy without causing severe side effects.

Phase: Phase I

Actively recruiting: No

Category: Children

ENCODE

The purpose of this investigation is to evaluate the impact of non-persistent and persistent chemical exposures on autoimmunity, gene expression and the microbiome in children with and without Crohn’s disease. If these chemical exposures are associated with differences in the epigenome and microbiome that are known to predispose for Crohn’s disease, the proposed research could further support changes in dietary behaviors to limit exposure, and efforts to regulate manufacturing processes that unintentionally and intentionally add these chemicals to food.

Phase: N/A

Actively recruiting: No

Category: Adults, Children, Healthy Controls

AA Genetic Study

The purpose of this study is to compare the genes of African American children and adults who have inflammatory bowel disease (IBD) with the genes of those who do not have IBD. Genes are pieces of hereditary material found in human cells. How an individual's genes cause intestinal inflammation in IBD is still being tested and not much is known about the association between genes and IBD in individuals with African ancestry. We hope to understand which genes cause IBD and if certain genes can help doctors personalize treatment for patients with IBD. For more information please contact IBDResearch@email.chop.edu.

Phase: N/A

Actively recruiting: No

Category: Adults, Children

Contrast-Enhanced Ultrasound Evaluation of Hypoxic Ischemic Injury

The goal of this HIE (Hypoxic Ischemic Encephalopathy) Study is to develop ultrasound techniques for detecting impaired blood supply to the brain using an investigational contrast agent. Our hope is to contribute to early diagnosis and improved outcomes.

Phase: N/A

Actively recruiting: No

Category: Children

ENERGY Study

TheENERGY study is for adults (ages 18 and up) with warm Autoimmune HemolyticAnemia (wAIHA) who are currently receiving treatment or have previously receivedtreatment. Thegoal of the study is to see if the study drug, Nipocalimab is safe andeffective in the treatment of wAIHA. Nipocalimab is an investigationalmedication designed to stop your immune system from destroying your red blood cells.

You may be able to join the study if you are:

18 years of age or older.
Diagnosed with wAIHA for at least 3months.
Currently receiving treatment orhave previously received treatment for wAIHA
Have platelet counts of more than30,000/ L and Hemoglobin less than 10 g/dL

Other studyrequirements will apply.

Phase: Phase II, Phase III

Actively recruiting: No

Category: Adults

Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism (for children under 1 year of age)

This study is arandomized, double-blind, placebo-controlled study that will evaluate thesafety and efficacy of dasiglucagon in children between the ages of 7 days and12 months who have congenital hyperinsulinism. Dasiglucagon will be given as asubcutaneous infusion while patients are admitted to the hospital. Othertreatments for hyperinsulinism may be changed as allowed while maintaining safeblood sugar levels. For the second part of the study, patients may be able togo home on the medication.

Phase: Phase II, Phase III

Actively recruiting: No

Category: Children

BMT CTN 1503 (STRIDE2): BMT for SCD

The purpose of this research study is to compare transplant (HCT/BMT) to standard of care treatment in young adults with SCD who have experienced complications. Once consented we would assess to see if you have a matched donor. Subjects who have a closely matched donor will undergo a bone marrow transplant. This is called the Donor Arm. Subjects who do not have a closely matched donor will receive the current standard of care for their disease. This is called the No Donor Arm. We will follow subjects in both arms for two years to evaluate outcomes over that time period.

Phase: Pilot, Phase II

Actively recruiting: No

Category: Adults, Children, MRI/Imaging Studies

Polyethylene Glycol Safety in Children

This study was designed to measure components or metabolites of PEG 3350 in blood and urine from children who were already taking PEG 3350 and to compare these levels to measurements of the same molecules from a group of children who were not taking PEG 3350. PEG 3350 was not prescribed as part of this study. Participants completed questionnaires and provided access to their medical records.Enrollment for this study has been completed. Laboratory analysis of specimens and data analysis are being conducted. This work will be submitted for publication once all analyses have been completed. When available, study results will be accessible on https://classic.clinicaltrials.gov/ct2/show/NCT05424757.

Phase: N/A

Actively recruiting: No

Category: Children, Healthy Controls

Aneurysms in ARPKD

The primary goal of this research study is to learn if patients with autosomal recessive polycystic kidney disease (ARPKD) are at risk for brain aneurysms or cardiovascular abnormalities. Participants in this study will involve patients that have a clinical diagnosis of ARPKD. The study will also involve a comparison group of healthy individuals that have no history of kidney or cardiac conditions.

Phase: Pilot

Actively recruiting: No

Category: Adults, Children, Healthy Controls, MRI/Imaging Studies, One-Time Visit Studies

CureGN Study

Cure Glomerulonephropathy Network (CureGN) is a multi-year study of 2,400 children and adults with four rare glomerular (kidney) diseases at over 50 centers in the US, Canada, and Italy. The purpose of CureGN is to create a patient research network to learn about four major causes of glomerular disease : 1. Minimal Change Disease (MCD) 2. Focal Segmental Glomerulosclerosis (FSGS) 3. Membranous Nephropathy (MN) 4. IgA Nephropathy (IgAN)

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children

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